Abstract
Standard treatment for high-grade glioma involves surgical resection followed by radiation therapy and temozolomide. Unfortunately, there are no standard treatment recommendations after recurrence and new therapies are needed for patients whose tumor recurs after first-line treatment. This single-arm, two-stage, interventional Phase II study evaluated the efficacy and safety of a combination of antineoplastons A10 and AS2-1. Nineteen patients were enrolled in the study (safety population), but fifteen patients with a median age of 9.4 years who met eligibility criteria were evaluated. The majority of subjects (12/15) were Caucasian and 8/15 (53%) were female. More than half (53%) of patients were diagnosed with glioblastoma and 33% with anaplastic astrocytoma. All patients had failed standard therapy including surgery, radiation, and chemotherapy. Antineoplastons were administered intravenously every four hours (median dose of A10 6.9 g/kg/d and AS2-1 0.30 g/kg/d) until objective response was documented and thereafter for a further 8 months. Clinical evaluations were performed every 8 weeks. All patients enrolled in the study were included in the safety analysis but only patients fulfilling the inclusion criteria were included in the efficacy evaluation. The duration of treatment with antineoplastons ranged from 2 weeks to 120 weeks. A complete response was documented in 2/15 (13%), partial response in 2/15 (13%), stable disease in 3/15 (20%). Progression-free survival at six months was 47% and overall survival (OS) at one year was 33.3%. One patient (6.7%) survived 10 years from treatment start. A small group of patients suffered reversible Grade 3 and 4 toxicities including hypernatremia 2/19 (11%) and decrease of neutrophils 1/19 (5%). There were no chronic toxicities. There was improvement of quality of life in patients who had objective response. It is concluded that antineoplastons show efficacy with an acceptable profile in this cohort of patients with recurrent high-grade glioma.
Highlights
While research carried out in recent years has brought exciting new advances in the field of neuro-oncology, this still needs to be translated into an improvement in clinical results [1]
The standard of care for newly diagnosed high-grade glioma (HGG) is surgical resection followed by daily temozolomide (TMZ) and radiation therapy (RT) with subsequent adjuvant TMZ [2] [3]
The reported results from studies with currently available treatments are even more disappointing in pediatric HGG, and very little progress has been made over the past decade regarding the introduction of effective therapeutic regimens [4]
Summary
While research carried out in recent years has brought exciting new advances in the field of neuro-oncology, this still needs to be translated into an improvement in clinical results [1]. The standard of care for newly diagnosed high-grade glioma (HGG) is surgical resection followed by daily temozolomide (TMZ) and radiation therapy (RT) with subsequent adjuvant TMZ [2] [3]. There are no standard therapy recommendations after recurrence [3]. The reported results from studies with currently available treatments are even more disappointing in pediatric HGG, and very little progress has been made over the past decade regarding the introduction of effective therapeutic regimens [4]. HGG in children are relatively uncommon and constitute approximately 17% of all pediatric brain tumors [5]. In children below 15 years of age GBM constitute only 2.8% of all central nervous system (CNS) tumors [5]
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